The plasma industry has experienced a strong growth in recent years, going from just over $5 billion in global sales in 2000 to almost $20 billion in 2015. As new indications for existing products are approved by the regulatory authorities and new plasma proteins are introduced, the plasma industry is expected to continue expanding. Some of the current research efforts with plasma-based products are described below:
New Products Fractionated from Plasma
Hundreds and possibly thousands of proteins exist in human plasma. A few of those proteins are under clinical investigation as new therapeutic products. These include: reconstituted HDL for acute coronary risk, plasmin for peripheral blood clots, ADAMTS-13 for Thrombotic Thrombocytopenic Purpura, Protein S for Protein S Deficiency, Factor II, V, and X for their respective coagulation deficiencies, Factor H for aHUS and plasminogen for Ligneous Conjunctivitis.
New Indications for Plasma-based Products
Other plasma-based products are under investigation for additional diseases or indications, including:
- IVIG in various neurological diseases (Multiple Sclerosis, Neuropathic Pain, Chronic Fatigue Syndrome, Post-Polio Syndrome, Dermatomyositis, etc.),
- Albumin in Stroke, Cirrhosis, Alzheimer’s Disease, Malaria and Sepsis,
- Alpha-1 antitrypsin in lung Graft-vs-Host Disease (GVHD), Lung injuries, and Type 1 Diabetes, and
- Fibrinogen in aortic aneurysm surgery with fibrinogen deficiency,
The first recombinant protein used as an alternative to a plasma-based product was recombinant factor VIII, which was commercialized in the early 1990’s. Since then, many recombinant proteins have been developed, including recombinant factor VIIa, factor IX, factor XIII, von Willebrand factor, albumin, antithrombin-III, C1 esterase inhibitor (C1-INH) and thrombin. Additional recombinant proteins are under development including recombinant fibrinogen, alpha-1 antitrypsin, plasmin and others.
In 2014, the first extended half-life recombinant factor VIII and IX products were approved for sale. This marked the first recombinant products, which had improved bio-availability over their plasma-derived competitors. By extending their half-life, these products feature higher trough levels in hemophilia patients, allowing them to infuse the product less frequently, which is more convenient for patients. The introduction of these new recombinant products has increased the number of products available to patients in recent years, leading to price erosion of the less advanced products, including the plasma-derived. In the future, non-factor protein-based therapies such as bi-specific antibodies and gene therapy are likely to result in further competition in the field.
In addition to new proteins and indications, the plasma industry continues to investigate new formulations for plasma-based products. For example, the original intramuscular formulation of immune globulins was not able to provide the volume necessary for optimal dosing in disease treatment. The development of intravenous (IVIG) formulations at 5%, then 10% concentration allowed for larger quantities of immune globulin to be infused, expanding the scope of diseases which could be successfully be treated with the product. More recently, subcutaneous (SCIG) preparations have gained market acceptance. So far, they have been mostly used for low dose applications, but research continues to push the boundaries to additional diseases. Smaller vial sizes have made it possible to administer coagulation factors to small infants, avoiding the need for ports, and efforts are made to find ways to administer these products through different routes. New formulations are in development for other plasma-derived products, such as inhaled A1PI or subcutaneous C1-INH, which may increase their usefulness and/or decrease administration costs.
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Since 1974, the Marketing Research Bureau has been supplying market data and intelligence to the plasma industry. Its syndicated reports cover over 70 countries, and provide quantitative and qualitative data. Other studies forecast the demand for the main plasma proteins and other issues. For more information, please contact us or give us a call.